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Regulus Presents Positive Preclinical Data Demonstrating that microRNA-21 Plays an Important Role in Alport Syndrome
Date:11/8/2013

LA JOLLA, Calif., Nov. 8, 2013 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that positive new preclinical data demonstrating that microRNA-21 (miR-21) plays an important role in Alport Syndrome will be presented at the American Society of Nephrology's Kidney Week 2013 meeting being held November 5-10 in Atlanta, GA.

In a poster presentation titled "Identification of the Pathologic Role of miR-21 in Alport's Kidney Disease" on Friday, November 8, 2013 from 10:00 a.m. EST to 12:00 p.m. EST, Regulus and Genzyme, a Sanofi company, presented new preclinical data demonstrating that miR-21 plays an important role in the disease progression of Alport Syndrome in collagen 4A3 deficient mice.  The study assessed urine albumin, kidney function, pathology and gene expression in the mice at 4, 6, 9, 12 and 15 weeks of age.  Efficacy of an anti-miR-21 candidate was assessed in mice starting at 5 weeks of age.  Urine albumin, renal function, kidney pathology and lifespan were used as efficacy endpoints.  Treatment with an anti-miR-21 candidate significantly improved renal function, significantly reversed regulated genes and pathways associated with renal pathology, and increased the lifespan of the mice by 20 percent. The poster can be accessed on Regulus' website, http://www.regulusrx.com.

"We are extremely pleased with the results from this study and believe these positive data validate that treatment with an anti-miR-21 candidate may be an important new therapy for patients with Alport Syndrome," said Neil W. Gibson, Ph.D., Chief Scientific Officer at Regulus. "These data bring us one step clo
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